A Phase 1 Randomized, Blinded, Placebo-Controlled, Single- and Multiple-Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics, With an Open-Label Target Occupancy Study of BIIB113 in Healthy Participants

Parts A and B: The primary objective of this study is to evaluate the safety and tolerability of single and multiple ascending oral doses of BIIB113 in healthy participants. The secondary objectives of this study are to evaluate the single and multiple oral dose pharmacokinetic (PK) profile of BIIB113 in healthy participants and to evaluate the effect of food on the single oral dose of BIIB113 in healthy participants of Part A cohort 3.
Part C: The primary objectives of this study are to evaluate the safety and tolerability of single and multiple ascending oral doses of BIIB113 in healthy participants and to determine target occupancy (TO) as measured by O-GlcNAcase-Positron Emission Tomography (OGA-PET) of single and multiple oral doses of BIIB113 in healthy participants.

开始日期2022-01-24

申办/合作机构

Biogen, Inc.

100 项与 BIIB-113 相关的临床结果

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100 项与 BIIB-113 相关的转化医学

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100 项与 BIIB-113 相关的专利（医药）

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项与 BIIB-113 相关的文献（医药）

2025-09-01·JPAD-Journal of Prevention of Alzheimers Disease

Results of the first-in-human, randomized, double-blind, placebo-controlled, single- and multiple-ascending dose study of BIIB113 in healthy volunteers

Article

作者: Nery, Flavia C ; Wilkes, Denisa ; Landen, Jaren ; Arnold, H Moore ; Yachnin, Jeffrey ; Wu, Shuang ; Suttle, Ben ; Bullain, Szofia ; Hirschhorn, Beth ; Jones, Lori ; Varrone, Andrea ; Curiale, Gioacchino ; Nag, Sangram ; Gallagher, Diana ; Stenkrona, Per ; Xie, Jing ; Singh, Dave ; Bolin, Martin ; Halldin, Christer ; Yesilalan, Esin ; Kaliszczak, Maciej ; Morén, Anton Forsberg ; Hering, Heike

BACKGROUND:

Preclinical studies have demonstrated that inhibition of the O-linked β-N-acetylglucosaminidase enzyme increases tau O-linked β-N-acetylglucosaminylation and may attenuate tau pathology in Alzheimer's disease.

OBJECTIVES:

To examine the safety, tolerability, pharmacokinetics, and target occupancy of single- and multiple-ascending oral doses of the small-molecule O-linked β-N-acetylglucosaminidase inhibitor, BIIB113.

DESIGN:

Study 276HV101 was a first-in-human, multicenter, Phase 1, randomized, double-blind, placebo-controlled, single- and multiple-ascending dose trial.

SETTING:

72 participants were enrolled from February 2022 through July 2023.

PARTICIPANTS:

Adult healthy female and infertile/vasectomized male participants.

INTERVENTION:

In the single-ascending dose substudy, participants received a single dose of placebo or BIIB113 0.5, 3, 15, or 50 mg. In the 14-day multiple-ascending dose substudy, participants received placebo or BIIB113 15 or 50 mg once daily. In the target occupancy substudy, participants received either a single dose of BIIB113 0.5 or 3 mg or a once-daily dose of BIIB113 0.5 mg.

MEASUREMENTS:

Safety and tolerability were measured by incidence of adverse events. Pharmacokinetic and concentration-time profiles were assessed. Target occupancy was evaluated using the carbon-11 BIO-1819,578 radioligand.

RESULTS:

BIIB113 was generally well tolerated. Pharmacokinetics were linear over the dose range, with a half-life of approximately 30 h. Administration with food decreased the rate but did not affect the extent of absorption. There were no clinically meaningful differences in pharmacokinetics between elderly and nonelderly participants. Multiple once-daily doses of BIIB113 0.5 mg maintained a target occupancy of ≥90 % up to 48 h.

CONCLUSIONS:

BIIB113 was well tolerated and achieved high levels of target occupancy.

项与 BIIB-113 相关的新闻（医药）

2025-03-05

·Pharmaceutical Technology

Asceneuron halts Alzheimer’s trial adding to tau-targeting setbacks

A build-up of the abnormal tau can contribute to Alzheimer’s disease symptoms. Credit: Andrew Brookes via Getty Images. After Eli Lilly and Biogen’s setbacks in developing O-GlaNAcase (OGA) inhibitors for Alzheimer’s disease, Asceneuron has now halted its own Phase II trial of ASN51, its experimental oral OGA inhibitor. The company disclosed the decision in an update to the federal clinical trials database ClinicalTrials.gov , describing it as “strategic” without providing further details. The trial, which kicked off in October 2024, was expected to run for two years. The study (NCT06677203) was set to evaluate two doses of ASN51 against a placebo in 123 patients with early Alzheimer’s, focusing on safety, tolerability, suicide severity assessment through the Columbia-Suicide Severity Rating Scale (C-SSRS), and tau protein levels in cerebrospinal fluid. Asceneuron had previously said that ASN51 could be a disease-modifying therapy, with applications beyond Alzheimer’s in diseases such as Parkinson’s and amyotrophic lateral sclerosis (ALS). The decision to move ASN51 into this Phase II study was supported by a $100m Series C funding round announced in July 2024. The financing was led by Novo Holdings, the controlling shareholder of Novo Nordisk. Novo Holdings’ senior partner for venture investments Naveed Siddiqi described ASN51 as offering “the potential paradigm shift” in Alzheimer’s treatment at the time of the funding round. Asceneuron had already demonstrated in multiple Phase I studies that ASN51 reaches the nervous system and impacts the OGA enzyme. OGA inhibitors have been explored as a potential new class of disease-modifying treatments for Alzheimer’s and other neurodegenerative diseases due to their ability to target tau protein accumulation. Unlike amyloid-targeting therapies – such as Eisai and Biogen’s Leqembi (lecanemab) – that have dominated Alzheimer’s drug development, OGA inhibitors aim to slow disease progression by preventing the buildup of toxic tau aggregates in the brain. This termination follows setbacks for other OGA inhibitors in development. In August 2024, Eli Lilly disclosed that its OGA inhibitor, LY3372689, failed to meet the primary endpoint in a Phase II trial (NCT05063539) for early symptomatic Alzheimer’s. The study did not show a statistically significant benefit in slowing disease progression as measured by the integrated Alzheimer’s Disease Rating Scale (iADRS). Lilly also previously abandoned its antibody-based tau inhibitor zagotenemab in 2021 while Roche discontinued the development of semorinemab in January 2024. Last month, Biogen discontinued an early-stage OGA inhibitor programme, BIIB113. Beyond ASN51, Asceneuron has another OGA inhibitor, ASN90, which it licensed to Ferrer in February 2023. ASN90 is currently in a Phase II trial (NCT06355531) for progressive supranuclear palsy, a rare tau-related neurodegenerative disorder.

临床2期临床1期临床结果

2025-03-01

·思齐俱乐部

辉瑞、渤健接连削减研发管线！

新药研发风险高，投入大，回报周期长，早已让药企砍管线成为常态。而近年来，出于资源配置和资金聚集的问题，医药企业的降本大刀更是频频挥向新药研发管线。近期，辉瑞、渤健、礼来等跨国药企就已相继终止了多款高价新药的开发。近日，有报道称，辉瑞宣布将终止B型血友病基因疗法Beqvez的开发和商业化。这款定价高达350万美元的基因疗法，从获批到被放弃，仅仅过了一年的时间。公开资料显示，该疗法的研发始于Spark Therapeutics，辉瑞在2014年从Spark获得了Beqvez的授权。2024年1月，Beqvez获得加拿大卫生部批准上市，用于治疗18岁或以上中重度至重度血友病B成人患者；2024年4月，Beqvez获得美国FDA批准上市。据悉，放弃Beqvez后，辉瑞打算将其资源集中在其认为将对患者产生重大影响的治疗方法上，例如近期获得批准的一种血友病A新药Hympavzi上。Hympavzi是一种抗组织因子途径抑制剂(TFPI)。 2月12日，渤健(Biogen)宣布终止4款神经类药物的开发，包括针对糖尿病周围神经性疼痛的BIIB143(cemdomespib)、针对早期阿尔茨海默病的BIIB113、针对早期帕金森的BIIB094和针对多系统萎缩症的BIIB101。在此之前，礼来也透露，在2024年第四季度，其已对两个主要治疗领域的研发管线进行优化，终止了处于临床阶段的阿尔茨海默病和肥胖症候选药物项目。其中，礼来的口服O - GlcNAcase抗tau蛋白药物cepemgnastat在一项2期试验中未能减缓早期症状性阿尔茨海默病患者的临床衰退，数月后，就已从研发管线中被移除。除了跨国药企，近年来还有上海医药、华东医药、科兴制药、亚虹医药、天坛生物、天士力等众多国内药企也先后终止了在研项目。其中，多数管线被叫停的原因包括临床试验结果未达预期、缺乏市场竞争力，以及外部环境变化导致的药品需求下降等。业内表示，这些药企终止药物开发的案例，反映出药企在新药开发和商业化上一直都在面临重大挑战。未来，在政策的引导和市场竞争加剧的情况下，药企对于管线的管理需更加理性、谨慎。来源：制药网点击阅读原文了解更多

上市批准基因疗法临床2期

2025-02-18

·医药健闻

艾伯维宣布其CEO接任董事长；南微医学完成跨国收购；百洋医药获罗氏美罗华在华独家推广权 | 日报

全球医疗行业每日重点资讯文 | 苏丁企业动态制药公司艾伯维(AbbVie)宣布任命其CEO罗伯特·迈克尔(Robert Michael)接替理查德·冈萨雷斯(Richard Gonzales)担任这家的董事长，自7月1日起生效。自2013年公司成立以来一直担任董事长的冈萨雷斯将于7月1日与长期担任公司首席独立董事的格伦·蒂尔顿(Glenn Tilton)一起从董事会退休。冈萨雷斯自2013年艾伯维公司从雅培独立出来之后一直担任公司CEO，去年方才卸任并将CEO职位交给迈克尔。迈克尔自艾伯维公司成立以来也一直在公司工作。史赛克(Stryker)宣布完成一项大规模债务发行，共筹集30亿美元。此次发行包括四系列债券，到期日从2027年到2035年不等，利率从4.550%到5.200%。外媒报道，此次财务举措是史赛克为即将收购Inari Medical 的战略提供资金的一部分，就在2025一开年，史赛克就公开宣布将以49亿美元收购Inari Medical。渤健（Biogen）在2024年财报中，宣布将停止4款中枢神经系统药物的开发工作。被砍掉的4款神经类药物管线，分别为：针对早期阿尔茨海默病的BIIB113、针对早期帕金森的BIIB094、针对多系统萎缩症的BIIB101、以及针对糖尿病周围神经性疼痛的BIIB143（cemdomespib）。渤健的开发主管Priya Singhal解释称，将研发资源集中于少数几个临床阶段的项目，相信具有较高的确定性，能够定期提供关键的研究成果，并有可能实现产品的市场推出。国产内镜龙头南微医学与Creo Medical在西班牙马德里正式完成对Creo Medical子公司CreoMedical S.L.U.（CME）51%股权的收购交割程序。南微医学通过全资子公司Micro-Tech (NL) International B.V.，成功收购Creo Medical Group plc持有的CME 51% 股权，让CME正式成为南微医学的控股子公司。此次收购使用使用自有资金不超过3,672万欧元。Creo Medical，总部位于英国，专注于外科内窥镜领域微创手术设备的开发和商业化。CME公司是Creo的全资子公司，是医疗器械产品销售的渠道公司。问止中医科技近日向港交所递交招股书，拟香港主板挂牌上市。如成功上市，问止中医将成为“中医+AI”领域第一股。问止中医成立于2018年，是一家由人工智能赋能的中医医疗服务提供商，业务包括AI辅助的中医医疗服务、中医生活产品及中医大脑订阅服务。问止中医自主开发了中医AI辅助诊疗系统——中医大脑，其近九成收入来自线上。人福医药公告，公司重整方案已获批准，重整投资方案实施完成后，招商生科将成为人福医药实质第一大股东与控股股东。1月16日，人福医药发布公告，公司收到控股股东当代科技管理人通知，当代科技、招商局创新科技（集团）有限公司与当代科技管理人于当日签订《重整投资协议》，若上述重整计划实施完毕，人福医药亦将变更为央企控股。质肽生物近日再获亿元融资，领投方为国风投（无锡）生物科技基金，老股东爱美客、蓝驰创投持续加码。质肽生物其自主研发的口服GLP-1类多肽ZT006片（体重管理适应症和2型糖尿病适应症）于去年11月获批临床，将开展I期首次人体临床试验。复星医药在内部发布了PharmAID决策智能体平台。该平台充分利用了复星医药和水木分子ChatDD生物医药大模型，重点关注自动情报分析和商业价值评估等场景，助力研发人员和决策人员对海量实时变化行业信息的追踪效率，解决了商业评估客观性和标准一致性的问题，能多维度、全方位、高效支撑药物商业价值辅助决策。产业动态百洋医药宣布，与罗氏制药中国签订商业化合作协议，获得罗氏制药旗下肿瘤靶向药美罗华（通用名：利妥昔单抗）在中国内地地区的独家市场推广权。该药主要用于治疗非霍奇金淋巴瘤、慢性淋巴细胞白血病等疾病，于2000年4月在国内获批上市。美罗华2017年达到峰值销售额74亿瑞士法郎，由于2017年之后陆续出现生物仿制药，销售收入迅速下滑，2023年销售收入已下滑至16亿瑞士法郎。北京男性HPV疫苗接种工作已全面启动。默沙东方面表示，目前，北京地区疫苗供应量稳定，并已正式开通适龄男性预约接种HPV疫苗官方平台。价人乳头瘤病毒疫苗（酿酒酵母）的多项新适应证已获得国家药品监督管理局的上市批准，适用于9-26岁男性接种，这是中国境内首个且目前唯一获批、可适用于男性的HPV疫苗。 GSK宣布，美国FDA已批准其组合疫苗Penmenvy（MenABCWY）上市，在10-25岁人群中使用。这款疫苗针对脑膜炎球菌中最常见的5大血清群，它们通常是导致侵袭性脑膜炎球菌病（IMD）的原因。 Moderna表示，美国食品和药物管理局（FDA）在报告了一例罕见的神经系统副作用病例后，暂停了其实验性诺如病毒疫苗的后期试验。Moderna表示，这种被称为mRNA-1403的疫苗的试验在接到一例吉兰-巴雷综合征病例的不良事件报告后被临床暂停，该病例目前正在调查中。三生制药与四川百利天恒药业股份有限公司达成《战略合作协议》，双方将共同推进707和BL-B01D1联合用药，在中国大陆地区用于实体瘤的治疗，探索双抗+双抗ADC在肿瘤治疗上的未被发掘的潜在价值。707是依托三生制药CLF2专利平台开发的靶向VEGF/PD-1双抗，具有Best-in-class（同类最优）潜力。BL-B01D1是百利天恒自主研发的全球首创也是唯一进入临床阶段的靶向EGFR×HER3的双抗ADC。驯鹿生物宣布，中国香港卫生署已正式受理其CAR-T细胞产品伊基奥仑赛注射液（FUCASO）的新药上市许可申请（NDA)，用于治疗既往经过至少3线治疗后进展的复发/难治性多发性骨髓瘤（R/RMM）成人患者。这是香港卫生署首次正式受理国产CAR-T细胞产品的新药上市申请。由于伊基奥仑赛注射液已经实现为香港地区患者跨境供药，一旦成功获批，香港患者将可直接使用驯鹿生物南京生产基地制备的该产品。信达生物宣布其PD-1/IL-2α-bias双特异性抗体融合蛋白IBI363获得美国食品和药物监督管理局（FDA）授予快速通道资格，拟定适应症为抗PD-(L)1免疫检查点抑制剂及含铂化疗治疗后进展的局部晚期或转移性鳞状非小细胞肺癌。IBI363在多种晚期实体瘤中展现出了积极的疗效和安全性信号，目前信达生物正在中国和美国等同时开展IBI363的1/2期临床研究。由北京儿童医院与北京百川智能科技有限公司、小儿方健康科技（北京）有限公司联合研发的“AI儿科医生”在经过北京儿童医院近一个月的内测并得到专家认可后，日前正式“上岗”。基于Baichuan M1大模型打造的“AI儿科医生”，与来自耳鼻咽喉头颈外科、肿瘤外科、肿瘤内科等科室的13位知名专家并行会诊，并给出了与专家组会诊结果高度吻合的建议。第三批全国中成药采购联盟集中采购中选结果正式出炉，本次共有174个药品中选，涉及20个产品组、95个产品。共有超150家药企获得中选资格。其中广州白云山明兴制药、哈尔滨珍宝制药药品中选数最多，均有3个；其次是钓鱼台医药集团吉林天强制药、广东雷允上、河南福森药业、黑龙江金九药业等，均有2个药品中选。本次集采覆盖了临床用量大、竞争格局复杂的38个独家品种。联系美通社 +86-10-5953 9500 info@prnasia.com

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100 项与 BIIB-113 相关的药物交易

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